WO2005017098A3 - Compositions et methodes utilisant un arnsi, des composes amphipathiques et des polycations - Google Patents

Compositions et methodes utilisant un arnsi, des composes amphipathiques et des polycations Download PDF

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Publication number
WO2005017098A3
WO2005017098A3 PCT/US2003/025121 US0325121W WO2005017098A3 WO 2005017098 A3 WO2005017098 A3 WO 2005017098A3 US 0325121 W US0325121 W US 0325121W WO 2005017098 A3 WO2005017098 A3 WO 2005017098A3
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WO
WIPO (PCT)
Prior art keywords
amphipathic
polycationic compounds
delivering sirna
sirna
delivering
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US2003/025121
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English (en)
Other versions
WO2005017098A2 (fr
Inventor
David L Lewis
James E Hagstrom
Hans Herweijer
Sean D Monahan
Jon A Wolf
Paul M Slattum
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Arrowhead Madison Inc
Original Assignee
Mirus Corp
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Filing date
Publication date
Application filed by Mirus Corp filed Critical Mirus Corp
Priority to EP03814415A priority Critical patent/EP1679964A4/fr
Publication of WO2005017098A2 publication Critical patent/WO2005017098A2/fr
Publication of WO2005017098A3 publication Critical patent/WO2005017098A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/58—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids containing heterocyclic rings, e.g. danazol, stanozolol, pancuronium or digitogenin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/74—Synthetic polymeric materials
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Fertilizers (AREA)
  • Processing Of Solid Wastes (AREA)

Abstract

L'invention concerne une composition de faible toxicité, qui comprend un composé amphipathique et un polycation utiles pour le transport d'un ARNsi vers une cellule. La composition de l'invention peut être utilisée dans un processus destiné à transporter un ARNsi vers une cellule animale, en particulier une cellule de mammifère, dans un format multipuits.
PCT/US2003/025121 2003-07-17 2003-08-11 Compositions et methodes utilisant un arnsi, des composes amphipathiques et des polycations Ceased WO2005017098A2 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP03814415A EP1679964A4 (fr) 2003-07-17 2003-08-11 Compositions et methodes utilisant un arnsi, des composes amphipathiques et des polycations

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US10/621,760 2003-07-17
US10/621,760 US20040019008A1 (en) 2002-05-28 2003-07-17 Compositions and processes using siRNA, amphipathic compounds and polycations

Publications (2)

Publication Number Publication Date
WO2005017098A2 WO2005017098A2 (fr) 2005-02-24
WO2005017098A3 true WO2005017098A3 (fr) 2005-06-30

Family

ID=34193477

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2003/025121 Ceased WO2005017098A2 (fr) 2003-07-17 2003-08-11 Compositions et methodes utilisant un arnsi, des composes amphipathiques et des polycations

Country Status (3)

Country Link
US (1) US20040019008A1 (fr)
EP (1) EP1679964A4 (fr)
WO (1) WO2005017098A2 (fr)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7601367B2 (en) * 2002-05-28 2009-10-13 Mirus Bio Llc Compositions and processes using siRNA, amphipathic compounds and polycations
EP2123759B1 (fr) * 2004-02-06 2014-01-15 Thermo Fisher Scientific Biosciences Inc. ARN stabilisés en tant que contrôles de la transfection et réactifs silencieux
US20090280567A1 (en) * 2004-02-06 2009-11-12 Dharmacon, Inc. Stabilized sirnas as transfection controls and silencing reagents
KR101147147B1 (ko) * 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
US20060014289A1 (en) * 2004-04-20 2006-01-19 Nastech Pharmaceutical Company Inc. Methods and compositions for enhancing delivery of double-stranded RNA or a double-stranded hybrid nucleic acid to regulate gene expression in mammalian cells
CA2565685A1 (fr) * 2004-05-04 2005-12-15 Nastech Pharmaceutical Company Inc. Compositions et procedes pour l'amelioration de l'administration d'acides nucleiques dans des cellules et pour la modification de l'expression de genes cibles dans des cellules
US20060040882A1 (en) * 2004-05-04 2006-02-23 Lishan Chen Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
US20060035815A1 (en) * 2004-05-04 2006-02-16 Nastech Pharmaceutical Company Inc. Pharmaceutical compositions for delivery of ribonucleic acid to a cell
JP2008532480A (ja) * 2004-11-22 2008-08-21 ダーマコン， インコーポレイテッド 乾燥遺伝子サイレンシング組成物を有する装置及びシステム
US7935811B2 (en) * 2004-11-22 2011-05-03 Dharmacon, Inc. Apparatus and system having dry gene silencing compositions
US20060166234A1 (en) * 2004-11-22 2006-07-27 Barbara Robertson Apparatus and system having dry control gene silencing compositions
US7923207B2 (en) * 2004-11-22 2011-04-12 Dharmacon, Inc. Apparatus and system having dry gene silencing pools
US20060223777A1 (en) * 2005-03-29 2006-10-05 Dharmacon, Inc. Highly functional short hairpin RNA
KR20080061397A (ko) * 2005-10-14 2008-07-02 나스텍 파마수티컬 컴퍼니 인코포레이티드 Ｒｎａ 치료제용 펩티드 리보핵산 축합물 입자들의화합물들과 방법들
RU2451022C2 (ru) * 2005-12-15 2012-05-20 Сантр Насьональ Де Ля Решерш Сьентифик (Снрс) Катионные олигонуклеотиды, автоматизированные способы их получения и их применение
EP1844772A1 (fr) * 2006-04-06 2007-10-17 Polyplus-Transfection SA Compositions pour la transfection d'oligonucleotides pour le silençage génétique et applications biologiques et therapeutiques
US20070281900A1 (en) * 2006-05-05 2007-12-06 Nastech Pharmaceutical Company Inc. COMPOSITIONS AND METHODS FOR LIPID AND POLYPEPTIDE BASED siRNA INTRACELLULAR DELIVERY
US20070275923A1 (en) * 2006-05-25 2007-11-29 Nastech Pharmaceutical Company Inc. CATIONIC PEPTIDES FOR siRNA INTRACELLULAR DELIVERY
AU2007286059A1 (en) * 2006-08-18 2008-02-21 Mdrna, Inc. Dicer substrate RNA peptide conjugates and methods for RNA therapeutics
WO2008036841A2 (fr) * 2006-09-22 2008-03-27 Dharmacon, Inc. Complexes d'oligonucléotides tripartites et procédés de silençage de gènes par interférence arn
FR2926818B1 (fr) * 2008-01-30 2012-04-06 Centre Nat Rech Scient siRNA CATIONIQUES, SYNTHESE ET UTILISATION POUR L'ARN INTERFERENCE
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
WO2011106688A1 (fr) * 2010-02-26 2011-09-01 Catabasis Pharmaceuticals, Inc. Conjugués de bis-acide gras et leurs utilisations

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002010177A1 (fr) * 2000-08-01 2002-02-07 Gmp Companies, Inc. Sels ammonium d'inositol hexaphosphate et leurs utilisations
US20030044983A1 (en) * 1999-11-12 2003-03-06 Hans Herweijer Nucleic acid transfer complexes

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US5744335A (en) * 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
US20030143204A1 (en) * 2001-07-27 2003-07-31 Lewis David L. Inhibition of RNA function by delivery of inhibitors to animal cells
US20030092180A1 (en) * 2001-08-27 2003-05-15 David Lewis Inhibition of gene expression by delivery of small interfering RNA to post-embryonic animal cells in vivo
JP2001500162A (ja) * 1997-06-12 2001-01-09 トランジェーヌ、ソシエテ、アノニム 少なくとも１種の治療上有効な物質、特にポリヌクレオチド、を標的細胞に導入するための新規脂質複合体および遺伝子治療における使用
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
DE60041978D1 (de) * 1999-11-15 2009-05-20 Biocure Inc Auf äussere bedingungen ansprechende polymere hohle partikel
US7101995B2 (en) * 2001-08-27 2006-09-05 Mirus Bio Corporation Compositions and processes using siRNA, amphipathic compounds and polycations
WO2003064666A1 (fr) * 2002-02-01 2003-08-07 Transgene S.A. Vecteurs adenoviraux destines a moduler les activites cellulaires associees aux pod (domaines oncogeniques pml)
US20040224405A1 (en) * 2003-05-06 2004-11-11 Dharmacon Inc. siRNA induced systemic gene silencing in mammalian systems

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030044983A1 (en) * 1999-11-12 2003-03-06 Hans Herweijer Nucleic acid transfer complexes
WO2002010177A1 (fr) * 2000-08-01 2002-02-07 Gmp Companies, Inc. Sels ammonium d'inositol hexaphosphate et leurs utilisations

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
See also references of EP1679964A4 *

Also Published As

Publication number Publication date
US20040019008A1 (en) 2004-01-29
WO2005017098A2 (fr) 2005-02-24
EP1679964A2 (fr) 2006-07-19
EP1679964A4 (fr) 2009-06-24

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